カテゴリー: Research

Start of recruitment for the ATTUNE clinical trial

The ATTUNE trial is a Phase 1/2 clinical study sponsored by Ionis. It aims to evaluate ION440, an investigational Antisense Oligonucleotide (ASO) therapy designed to address the root cause of MECP2 Duplication Syndrome (MDS).

Recruitment starts for HERO: CRISPR cas13-clinical trial for MDS

The HERO clinical trial is a pioneering study evaluating HG204, a novel CRISPR Cas13 RNA-editing therapy, for MECP2 Duplication Syndrome (MDS). Sponsored by HuidaGene, this trial marks an important step in the search for a treatment for MDS. In November 2024, the first patient was successfully administered HG204, offering renewed hope to the MDS community.

ION440: IONIS brings MDS community to the next level

Written by David and verified by Ionis Following Ionis’ recent announcement about the upcoming clinical trial, we aim to provide a clearer understanding of this trial. It is especially important for families considering enrolling their child to know what a clinical trial is, particularly in the context of rare diseases like MECP2 Duplication Syndrome (MDS). A clinical trial is a research study that investigates new potential treatments, assessing their safety and effects on humans. Clinical trials are often conducted as part of a larger clinical development program. A clinical development program is a series of clinical trials conducted in progressive phases (i.e., Phase 1-3) designed to determine whether new potential […]

HuidaGene: Moving towards clinical trials

In February 2024, David Covini, President of DupMECP2, visited the innovative biotech company HuidaGene Therapeutics in Shanghai. The visit provided an exciting glimpse into the future for a treatment for MECP2 duplication syndrome (MDS). Led by Dr. Alvin Luk, CEO at HuidaGene, the Company is at the forefront of developing a groundbreaking CRISPR technology that could revolutionize the lives of children affected by MDS.  The visit was an incredible opportunity to witness firsthand the cutting-edge facilities and the creative environment that fosters such innovative research. The office space even includes a climbing wall, promoting a dynamic and creative work atmosphere. Alvin and his Medical Director of MDS, Dr. Gloria Tang, […]

Huidagene: A promising CRISPR-CAS13 technology from Shanghai (China)

Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders. Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome, HG204 aims to regulate the overproduction of the MeCP2 protein levels by inducing the degradation of overexpressed MECP2 RNA. This innovative drug candidate holds the potential to cure MDS through a single administration. Our organization has had productive discussions with Huidagene’s leaders to learn more about this innovative technology and what it means for the future. Preclinical studies using HG204 in a […]